Cell transplant reverses blindness.

December 17, 2001.
BBC News.

UK scientists have developed a pioneering technique that could lead to a treatment for an eye disease that affects older people.

Experts used genetic engineering to restore vision to rats, which went blind soon after birth, and hope future clinical trials in humans could pave the way for a treatment for age-related macular degeneration (AMD).

This work represents a tremendous leap forward

Institute of Ophthalmology Researchers from the Institute of Ophthalmology in London joined forces with colleagues in Sheffield and the USA to transplant human retinal pigment epithelial (RPE) cells into the rats, which were born with a genetic predisposition to retinal degeneration and sight loss.

RPE cells support the retina and are essential components of vision, but become dysfunctional in people with AMD, which is the leading cause of sight loss in people aged over 50 in the western world.

It is the first time genetically engineered RPE cells, grown in a test tube, have been successfully transplanted into another living creature.

Not only did the new cells survive, they restored sight to the rats, which were able to navigate and respond to visual stimuli.

The experiment's results, published in the journal Nature Neuroscience, raise the possibility that such a treatment could be used in the future to help patients suffering from such retinal degeneration as AMD.

We are very excited by the results of this research

Royal National Institute for the Blind Professor John Greenwood from the Institute of Ophthalmology said: "The transplanting of genetically engineered RPE cells is totally unique.

"At present, there is no truly effective treatment or cure for age-related macular degeneration.

"We believe this work represents a tremendous leap forward in our endeavour towards developing a feasible strategy for treating patients with this debilitating disease."

Major threat

Over 500,000 people in the UK have lost their sight due to AMD and the number at risk and with serious visual disability is much greater.

The Royal National Institute for the Blind (RNIB) welcomed the development. An spokesman said: "We are very excited by the results of this research.

"We welcome any scientific advances towards replacement or regeneration of sight, but it's very important for people to be aware that this research is at an early stage."

It is hoped the research may lead directly to new surgical treatments for AMD aimed at the replacement of diseased RPE.

Human clinical trials of any new treatments are expected to be carried out in the future at Moorfields Eye Hospital in London.